Abu Dhabi performs the world’s first to deliver ITVISMA for spinal muscular atrophy

Abu Dhabi just flipped a major page in medical history. The Emirate became the first place in the world to administer a breakthrough gene replacement therapy called ITVISMA for spinal muscular atrophy (SMA), a rare genetic neuromuscular condition.

What makes this moment striking is not just the novelty of the treatment, but the sheer ambition behind it — a small city-state pushing the boundaries of precision medicine and global health leadership.

How did it work?

At its heart, gene replacement therapy is about fixing what's broken at the source. Instead of managing symptoms, it hones in on the root genetic cause of a disease. In the case of ITVISMA (onasemnogene abeparvovec), the therapy replaces the missing or nonfunctioning SMN1 gene, a gene critical for producing the survival motor neuron protein that keeps muscles working.

In lay terms, here is the gist:

• A one-time therapy means a single administration, not lifelong infusions.
• It's targeted gene replacement.
• Designed for patients 2 years and older with a confirmed mutation of SMN1.

Dr. Noura Khamis Al Ghaithi, Under-Secretary of the Department of Health - Abu Dhabi, summed up the scientific leap this way: "This milestone reflects Abu Dhabi's commitment to delivering world-class care and strengthening its position as a global leader in healthcare driven by genomics and precision medicine."

Why does it matter?

This isn't just another clinical first. It's a pivot point in how we treat genetic disease. For decades, conditions like spinal muscular atrophy meant constant clinic visits, complex care plans, and heavy burdens on families. Gene replacement therapy changes that narrative. By addressing the underlying genetic defect, it offers the potential to dramatically improve motor function and reduce the need for lifelong treatments.

Here's what's on the table:

• A chance for patients to get real gains in quality of life.
• A shift from reactive care to proactive correction.
• A model of treatment that guides future rare disease care.

Bader Al Qubaisi, CEO at Sheikh Khalifa Medical City, put it plainly: "Delivering the world's first ITVISMA treatment at SKMC is a testament to Abu Dhabi's integrated healthcare ecosystem." His words echo a deeper truth: this is not just a medical feat but a healthcare system demonstrating muscle and maturity.

The context

The UAE approved ITVISMA for use on November 25, 2025, making the country one of the first in the world after the United States to embrace this therapy. Sheikh Khalifa Medical City, under the watch of the Department of Health - Abu Dhabi, carried out the first administration.

This achievement isn't isolated. Abu Dhabi is rapidly building an ecosystem that blends universal genetic screening, faster health technology reviews, and cutting-edge care pathways for rare conditions. Here, a newborn might be screened for hundreds of treatable genetic conditions, and a child might benefit from a single shot that alters the entire course of their life. It's ambitious, yes. But in the right ways.

In a world where healthcare innovation often feels stuck in incremental steps, Abu Dhabi just took a quantum leap. The question now isn't just whether other places will follow — it's how fast and how smart they will be when they do.